ARIAD Announces U.S. Food and Drug Administration Acceptance of NDA Filing for Brigatinib

ARIAD Pharmaceuticals (NASDAQ: ARIA) today announced that the U.S. Food and Drug Administration (FDA) has accepted for review the New Drug Application (NDA) for ARIAD’s investigational oral anaplastic lymphoma kinase (ALK) inhibitor, brigatinib, in patients with metastatic ALK-positive (ALK+) non-small cell lung cancer (NSCLC) who have progressed on crizotinib. The FDA granted ARIAD’s request for Priority Review and has set an action date of April 29, 2017 under the Prescription Drug User Fee Act (PDUFA).
“The FDA acceptance of our application is an important milestone in our ongoing efforts to discover, develop and deliver highly innovative treatments for patients with rare cancers,” said Paris Panayiotopoulos, president and chief executive officer of ARIAD. “We are pleased that our significant R&D investments in brigatinib and our work with the FDA are bringing us closer to potentially offering a treatment option for patients with ALK+ NSCLC who are refractory to crizotinib. We look forward to continuing to work closely with the FDA during the brigatinib NDA review and remain committed to developing critical therapies for unserved and underserved small patient populations suffering from rare
cancers.”
ARIAD’s NDA submission includes clinical data from its Phase 1/2 and pivotal Phase 2 ALTA trials of brigatinib. The FDA’s Priority Review status accelerates the review time from 10 months to a goal of six months from the filing of the application. Brigatinib received Breakthrough Therapy designation from the FDA for the treatment of patients with ALK+ NSCLC whose tumors are resistant to crizotinib, and was granted orphan drug designation by the FDA for the treatment of ALK-positive, ROS1-positive, and EGFR-positive NSCLC. ARIAD plans to submit a Marketing Authorization Application (MAA) for brigatinib to the European Medicines Agency (EMA) in early 2017.
ARIAD is a small, research-driven biotechnology company. ARIAD has invested more than $1.3 billion in R&D since the Company was founded. In
2015, ARIAD generated $119 million in total revenue and invested $171 million, or 143 percent of revenue, in R&D.
About Brigatinib
Brigatinib is an investigational, targeted cancer medicine discovered
internally at ARIAD. It is in development for the treatment of patients
with anaplastic lymphoma kinase positive (ALK+) non-small cell lung
cancer (NSCLC). The global Phase 2 ALTA trial, in patients with locally
advanced or metastatic ALK+ NSCLC who were previously treated with
crizotinib, is the primary basis for brigatinib’s initial regulatory
review. ARIAD has also initiated the Phase 3 ALTA 1L trial to assess the
efficacy and safety of brigatinib in comparison to crizotinib in
patients with locally advanced or metastatic ALK+ NSCLC who have not
received prior treatment with an ALK inhibitor. More information on
brigatinib clinical trials, including the expanded
access program (EAP) for ALK+ NSCLC can be found here.
About ALK+ NSCLC
Non-small cell lung cancer (NSCLC) is the most common form of lung
cancer, accounting for approximately 85 percent of the estimated 228,190
new cases of lung cancer diagnosed each year in the United States,
according to the American Cancer Society. Anaplastic lymphoma kinase
(ALK) was first identified as a chromosomal rearrangement in anaplastic
large-cell lymphoma (ALCL). Genetic studies indicate that chromosomal
rearrangements in ALK are key drivers in a subset of NSCLC patients as
well. Approximately three to eight percent of patients with NSCLC have a
rearrangement in the ALK gene.
About ARIAD
ARIAD Pharmaceuticals, Inc., headquartered in Cambridge, Massachusetts
is focused on discovering, developing and commercializing precision
therapies for patients with rare cancers. ARIAD is working on new
medicines to advance the treatment of rare forms of chronic and acute
leukemia, lung cancer and other rare cancers. ARIAD utilizes
computational and structural approaches to design small-molecule drugs
that overcome resistance to existing cancer medicines. For additional
information, visit https://www.ariad.com
or follow ARIAD on Twitter (@ARIADPharm).
Forward-Looking Statements
This press release contains forward-looking statements, each of which
are qualified in their entirety by this cautionary statement. Any
statements contained herein which do not describe historical facts,
including, but not limited to statements about the anticipated timing
for potential regulatory approval of brigatinib in the United States,
the potential for brigatinib to provide a new treatment option to ALK+
NSCLC patients, the Company’s plans to file for regulatory approval of
brigatinib with the EMA, and the Company’s ongoing clinical development
of brigatinib, are forward-looking statements that are based on
management’s expectations and are subject to certain factors, risks and
uncertainties that may cause actual results, outcome of events, timing
and performance to differ materially from those expressed or implied by
such statements. These factors, risks and uncertainties include, but are
not limited to, our ability to successfully commercialize and generate
profits from sales of our products; our ability to meet anticipated
clinical trial commencement, enrollment and completion dates and
regulatory filing dates for our products and product candidates and to
move new development candidates into the clinic; our ability to execute
on our key corporate initiatives; regulatory developments and safety
issues, including difficulties or delays in obtaining regulatory and
pricing and reimbursement approvals to market our products; competition
from alternative therapies; our reliance on the performance of
third-party manufacturers, specialty pharmacies, distributors and other
collaborators for the supply, distribution, development and/or
commercialization of our products and product candidates; the occurrence
of adverse safety events with our products and product candidates; the
costs associated with our research, development, manufacturing,
commercialization and other activities; the conduct, timing and results
of preclinical and clinical studies of our products and product
candidates, including that preclinical data and early-stage clinical
data may not be replicated in later-stage clinical studies; the adequacy
of our capital resources and the availability of additional funding; the
ability to satisfy our contractual obligations, including under our
leases, convertible debt and royalty financing agreements; patent
protection and third-party intellectual property claims; litigation and
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conditions, health care reform, prices and reimbursement rates; and
other risk factors detailed in our public filings with the U.S.
Securities and Exchange Commission, including our most recent Annual
Report on Form 10-K and subsequent Quarterly Reports on Form 10-Q.
Except as otherwise noted, these forward-looking statements speak only
as of the date of this press release and we undertake no obligation to
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not to place considerable reliance on the forward-looking statements
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